Lessons from preventive infectious disease vaccines

Preventive vaccines for infectious disease indications are regulated by the Office of Vaccines Research and Review, CBER, FDA. FDA-regulated biological products, including vaccines, must be safe, pure, potent, and manufactured consistently according to current Good Manufacturing Practices. Regulatory issues include common principles of vaccine production and quality control, lot release testing, vaccine preclinical data, and clinical development. For Phase 3 studies, the following should be addressed: the case definition of the disease that the vaccine is intended to prevent (piloted during Phase 2) and the sample size to support efficacy and safety considerations. Also during development, validation data for immunological and other critical assays, clinical lot consistency studies, and the possible need for bridging studies, e.g., to support manufacturing changes or extrapolation to different populations, should be addressed.